Trial of Penicillamine in Advanced Primary Biliary Cirrhosis

E. Rolland Dickson, Thomas R. Fleming, Russell H. Wiesner, William P. Baldus, C. Richard Fleming, Jurgen Ludwig, John T. Mccall

Research output: Contribution to journalArticlepeer-review

113 Scopus citations


A total of 227 patients with histologically advanced primary biliary cirrhosis entered a double-blind, randomized, controlled trial to determine whether penicillamine (1 g per day) was therapeutically effective; 111 patients received the drug, and 116 received placebo. The two groups were highly comparable at entry with regard to clinical, biochemical, and histologic features. Penicillamine therapy did not result in an overall improvement in survival as compared with placebo. Clinical symptoms and serial hepatic laboratory values reflected the progressive nature of the disease and were similar in both groups. There were no substantial differences between treatment groups in the morphologic features of sequential biopsy specimens. The development of major side effects led to permanent discontinuation of penicillamine in 22 per cent of the patients taking the drug. We conclude that penicillamine is not useful for patients with histologically advanced primary biliary cirrhosis. The trial is being continued in patients with early histologic disease whose better prognosis necessitates longer follow-up. (N Engl J Med 1985; 312: 1011–5.).

Original languageEnglish (US)
Pages (from-to)1011-1015
Number of pages5
JournalNew England Journal of Medicine
Issue number16
StatePublished - Apr 18 1985

ASJC Scopus subject areas

  • General Medicine


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